Particle
1
Patient with genetic disorder
More than 10,000 human diseases are caused by faults in single genes. While each condition is relatively rare, they affect millions of people around the globe
2
Millions of particles injected into patient’s bloodstream or affected tissues. The particles can be harmless viruses or nanoparticles that carry the gene editing molecules
Guide molecule
Cas9
Healthy Gene
3
Each particle holds
– A guide molecule that
finds the DNA to be changed
– A powerful enzyme that cuts the target DNA
– ‘Healthy’ DNA to mend the mutated gene
4
The particles carrying the gene editing molecules get into the affected cells and enter the nuclei where the DNA lies
Nucleus
Inside the cell nucleus the guide molecule moves along the patient’s DNA until it finds a match. The guide is designed to stick to the targeted region of genetic code, such as a mutated gene, and it is here that the edit will take place
Guide
molecule
Mutated
gene
Healthy
gene
Once the guide molecule finds its target the attached enzyme swings into action and snips the DNA in half. The cell detects the damage and sets about trying to mend it
The cell repairs the breakage by inserting ‘healthy’ DNA that is injected alongside the gene editing molecules
1
Patient with genetic disorder
More than 10,000 human diseases are caused by faults in single genes. While each condition is relatively rare, they affect millions of people around the globe
2
Millions of particles injected into patient’s bloodstream or affected tissues. The particles can be harmless viruses or nanoparticles that carry the gene editing molecules
Particle
3
Each particle holds
– A guide molecule that
finds the DNA to be changed
– A powerful enzyme that cuts the target DNA
– ‘Healthy’ DNA to mend the mutated gene
Guide molecule
Enzyme
Healthy DNA
4
The particles carrying the gene editing molecules get into the affected cells and enter the nuclei where the DNA lies
Nucleus
The gene editing process
Mutated
gene
Guide
molecule
Healthy
gene
Inside the cell nucleus the guide molecule moves along the patient’s DNA until it finds a match. The guide is designed to stick to the targeted region of genetic code, such as a mutated gene, and it is here that the edit will take place
Once the guide molecule finds its target the attached enzyme swings into action and snips the DNA in half. The cell detects the damage and sets about trying to mend it
The cell repairs the breakage by inserting ‘healthy’ DNA that is injected alongside the gene editing molecules